syndrome

Servier acquires potential treatment for Fragile X syndrome, the most common genetic cause of autism spectrum disorder

Servier acquires KER-0193, a potential treatment for Fragile X syndrome, developed by Kaerus Bioscience, a Medicxi companyKER-0193, a novel, orally bioavailable…

3 months ago

Ascentage Pharma Announces Global Registrational Phase III Study of Lisaftoclax for First-line Treatment of Patients with Higher-Risk Myelodysplastic Syndrome Cleared by US FDA and EMA

ROCKVILLE, Md. and SUZHOU, China, Aug. 17, 2025 (GLOBE NEWSWIRE) -- Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855), a global, commercial…

4 months ago

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

July 11, 2025 16:30 ET  | Source: Ultragenyx Pharmaceutical Inc. Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls…

5 months ago

Halia Therapeutics Completes Enrollment in Phase 2a Clinical Trial of HT-6184 for Myelodysplastic Syndrome (MDS)

LEHI, Utah, June 4, 2025 /PRNewswire/ -- Halia Therapeutics, a clinical-stage biopharmaceutical company pioneering therapies inspired by genetic resilience, today…

6 months ago